The 14-week trial will see 210 children between the ages of 3 and 17 treated at 21 clinical sites in the US, Australia and New Zealand. The clinical study of Cannabidiol in Children and Adolescents with Fragile X, or CONNECT-FX trial, is expected to produce topline results late in the second quarter.
Once it has the results, the company plans to meet with the U.S. Food and Drug Administration about submitting a New Drug Application in the second half of the year, which could potentially be approved in mid-2021.
“We are excited about the potential for Zygel, if approved by the FDA, to be the first drug ever indicated to treat the behavioral symptoms of Fragile X, and completion of enrollment in this trial is an important step toward that goal,” CEO Armando Anido said in a statement. “I want to thank everyone who participated in this achievement, particularly the patients in the study, their families, the clinicians involved in CONNECT-FX and their staff.”
The primary endpoint of the study is the change in behavior from a baseline level, measured using the Aberrant Behavior Checklist-Community FXS Specific social avoidance subscale.
The study will also evaluate patients’ Irritability subscale scores, socially unresponsive/lethargic subscale scores and track improvement in what’s known as clinical global Impression.
Zynerba noted that the trial has deliberately enrolled a more severely affected population than the group that participated in its Phase 2 FAB-C trial, in order to demonstrate the anticipated full range of efficacy of Zygel. For instance, the average baseline social avoidance subscore in the CONNECT-FX trial is 7.2, compared to 5.1 in FAB-C.
The mean age of the patients is 9.8 years and 75% are male, the company said.
Fragile X is a genetic condition caused by a mutation in the FMR1 gene. The mutation causes the X chromosome to appear constricted, or fragile, under a microscope, hence its name. Patients with Fragile X often have learning disabilities and other cognitive impairments.
Zygel, a patented CBD-formulated clear gel, is designed to provide controlled drug delivery into the bloodstream through the skin. Some recent studies have suggested that neuropsychiatric conditions such as Fragile X Syndrome are associated with a disruption in the endocannabinoid system, and CBD may improve certain core social and behavioral symptoms by modulating that system.
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